Bill Brazell wrote the best commentary on the FDAs decision not to approve tolvaptan in 2013. He links to it in this commentary. Follow the link. It is a do-not-miss editorial. Here he shares his thoughts on REPRISE.

When someone took this picture of my cousin Michael and me in September 1968, no one knew that there were already tiny cysts growing throughout our infant kidneys:

Michael and Me, 1968

...when he was just 35, PKD’s complications took Michael’s life, leaving his young son and daughter and countless shocked, grieving relatives and friends behind.

We had inherited polycystic kidney disease, or PKD, from our dads, who were siblings. As Michael and I grew older, the cysts grew larger. In 2002, when he was just 35, PKD’s complications took Michael’s life, leaving his young son and daughter and countless shocked, grieving relatives and friends behind. The pain of his absence reminds me that many thousands of American families have Michael-sized holes in their lives, too — often more than one.

By raising blood pressure, harming kidney function and increasing the risk of aneurysm, PKD shortens millions of lives around the world. PKD patients in Canada, the European Union, the United Kingdom, and Japan are able to take a drug called tolvaptan to slow the disease’s progression. PKD patients in the U.S., however, have no access to tolvaptan unless they are, like me, subjects of a study.

(Thanks to the generosity of Otsuka, the pharmaceutical company that has treated our PKD community wonderfully, I’ve been able to take tolvaptan for more than 12 years, for free, in exchange for my data — a trade I’ve been very happy to make. Tolvaptan makes me thirstier than I’ve ever been, and causes me to urinate more than anyone else I know. But I would be happy to take it for the rest of my life.)

So even as their fellow PKD patients in other countries are able to slow the growth of their cysts with the help of tolvaptan, Americans have to wait for the FDA to revisit a decision it made more than four years ago.

Recently the New England Journal of Medicine published the results of the REPRISE trial, which confirmed all earlier studies: Tolvaptan slows the rate of growth of the cysts by an average of 35% — sometimes more — and slows the decline in glomerular filtration rate, or GFR, more than placebo.

As I wrote on TheAtlantic.com four years ago, it was frustrating to sit in the room, helpless, as the FDA declined to approve tolvaptan the first time it had the chance. Some doctors on the panel opined that the risks of the drug outweighed its possible rewards. They cited possible liver damage, even though no one in the study had sustained permanent liver damage. A few subjects had experienced an increase in liver enzyme levels, which returned to normal when they stopped taking the drug. Meanwhile the rewards — an extra five years before kidney failure in some cases — were deemed insufficient by doctors who showed no interest in learning what a difference five years could make to a person with a family and a career.

It is my fervent hope that the FDA will finally agree with the EU, the UK, Canada, and Japan: tolvaptan works. It slows the progression of PKD significantly. And by making new doses of the drug contingent on healthy liver enzyme levels, as it promised to do four years ago and has promised again to do, Otsuka will keep tolvaptan’s users safe.

Let’s approve it, shall we?

Bill Brazell is a member of the PKD Foundation’s all-volunteer board of trustees. He does not pretend to speak for the board, but wrote the above based on his own experience.